Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

Completed · Phase 2

Conditions studied: Duchenne Muscular Dystrophy (DMD)

In brief

This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Key facts

Study ID

NCT02420379

Run by

Sarepta Therapeutics, Inc.

People needed

33

Starts

2015-06-30

Expected to finish

2018-12-17

Last updated by the study team

2021-01-25

Who can join

Age: 4 and older, up to 6. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male 4-6 years of age.
• Diagnosis of DMD, genotypically confirmed.
• Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks.
• Intact right and left biceps muscles or two alternative upper arm muscle groups.
• Parent that is willing to provide consent and comply with study procedures.

You may not qualify if…

• Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
• Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months.
• Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities.
• Presence of other clinically significant illness.

Where it is running

• Neuromuscular Research Center of Arizona — Phoenix, Arizona, United States
• Ronald Reagan UCLA Medical Center — Los Angeles, California, United States
• University of California, Davis Medical Center — Sacramento, California, United States
• Stanford University Medical Center — Stanford, California, United States
• University of Florida, Shands Hospital — Gainesville, Florida, United States
• Rare Disease Research Center — Atlanta, Georgia, United States
• Children's Hospital of Atlanta — Atlanta, Georgia, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• St. Louis Children's Hospital — St Louis, Missouri, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Shriners Hospital for Children — Portland, Oregon, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Seattle Children's Hospital — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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